Key Insights

Highlights

Success Rate

81% trial completion

Published Results

53 trials with published results (32%)

Research Maturity

96 completed trials (57% of total)

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 39/100

Termination Rate

13.2%

22 terminated out of 167 trials

Success Rate

81.4%

-5.2% vs benchmark

Late-Stage Pipeline

25%

42 trials in Phase 3/4

Results Transparency

55%

53 of 96 completed with results

Key Signals

53 with results81% success22 terminated

Data Visualizations

Phase Distribution

99Total
Not Applicable (10)
Early P 1 (1)
P 1 (34)
P 2 (12)
P 3 (33)
P 4 (9)

Trial Status

Completed96
Recruiting23
Terminated22
Unknown15
Active Not Recruiting5
Withdrawn3

Trial Success Rate

81.4%

Benchmark: 86.6%

Based on 96 completed trials

Clinical Trials (167)

Showing 20 of 20 trials
NCT05568719Phase 3Recruiting

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

NCT05611801Phase 3Recruiting

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

NCT05932914Not Yet Recruiting

Liver Biopsy Following Gene Therapy For Hemophilia

NCT07080905Phase 3RecruitingPrimary

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

NCT06379789Phase 1RecruitingPrimary

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

NCT06003387Phase 3RecruitingPrimary

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

NCT03961243Phase 1Not Yet RecruitingPrimary

Lentiviral FIX Gene Therapy

NCT03938792Phase 3Completed

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

NCT04628871TerminatedPrimary

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

NCT03307980Phase 2CompletedPrimary

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

NCT05568459Active Not RecruitingPrimary

A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy

NCT05164471Phase 1TerminatedPrimary

Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B

NCT06399289Phase 3CompletedPrimary

Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy

NCT06349473Phase 1Recruiting

A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)

NCT05145127Phase 3Recruiting

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

NCT03655223Active Not Recruiting

Early Check: Expanded Screening in Newborns

NCT06611436Phase 1TerminatedPrimary

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

NCT07644832Phase 1Recruiting

An Open-label, Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic/Pharmacodynamic (PK/PD) Characteristics of SR604 Injection in Patients With Hemophilia A/B and Congenital Factor VII Deficiency

NCT03861273Phase 3Active Not RecruitingPrimary

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

NCT05360706CompletedPrimary

Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B

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